Agios' mitapivat sickle cell decision slips to February 2027 as sales reach $55.4m

Agios Pharmaceuticals will have to wait until 1 February 2027 to learn whether the US regulator will let mitapivat into sickle cell disease. The company disclosed the new goal date on 5 November 2026, alongside its third-quarter results. The extra three months push back any revenue from a second major indication for the drug.

Agios said the FDA extended the timeline after deciding that its answer to an agency question counted as a major amendment to the supplemental application. The question was whether a risk evaluation and mitigation strategy (REMS) is needed for mitapivat in sickle cell disease. The information on file does not say whether the FDA has concluded that a REMS is required.

By way of context, the FDA can lengthen a review clock, typically by three months, when a sponsor submits a major amendment during review, so that it has time to assess the new material.

The delay comes as the company's near-term sales rest on thalassaemia. Agios booked net revenue of $55.4 million from mitapivat, sold as PYRUKYND and AQVESME, in the quarter. It attributed the figure mainly to the US launch of AQVESME in thalassaemia.

Agios ended the period with $907.0 million in cash and a quarterly net loss of $65.0 million. It also said it is cutting research and development headcount by more than 100 roles.